In an ongoing Phase 2 trial, participants in a cohort receiving a 10-milligram dose of PGN-EDODM1 showed mean splicing correction of about 47%, the highest level reported so far in a multiple-dose study, PepGen said. The company also reported improvements across several functional measures, including hand grip strength and a walking and running test.

PepGen said it is advancing the program to its third and highest-dose cohort, which is fully enrolled. “We are very confident in PGN-EDODM1’s potential to deliver meaningful benefit to the DM1 community,” Chief Executive James McArthur said.

Myotonic dystrophy type 1 is a rare, progressive genetic disease affecting muscles and multiple body systems. The company estimates it affects more than 110,000 people in the U.S. and the E.U. Average life expectancy for people with non-congenital DM1 is about 50 to 60 years, PepGen said.

The trial update came alongside PepGen’s third-quarter results. The company reported a loss of $19.4 million, or 28 cents a share, compared with a loss of $18 million, or 52 cents a share, a year earlier. FactSet analysts had expected a loss of 27 cents a share.